Process and strategies for patient engagement and outreach in the Sickle Cell Disease (SCD) community to promote clinical trial participation.

INTRODUCTION: Sickle cell disease (SCD) is the most common inherited blood disorder in the United States. The Cure Sickle Cell Initiative (CureSCi) was created by the National Heart, Lung and Blood Institute (NHLBI) to improve the lives of people with SCD by accelerating the advancement of safe and promising genetic therapies, engaging the SCD community and healthcare providers, and encouraging collaboration among stakeholders. CureSCi is a collaborative, patient-focused research effort that includes patients at every level of the Initiative. Patient engagement is a key component, particularly during the development of clinical trials. OBJECTIVE: To describe the processes and strategies utilized by the CureSCi to engage individuals living with SCD about genetic therapy clinical trials. METHODS: The CureSCi Patient Engagement team conducted nine Community Outreach visits in 2019, each of which was structured to include separate group interviews with patients and medical providers. A set of guidance questions was developed for each audience and the interviews lasted approximately 60 minutes. RESULTS: Patients were asked about their participation in a clinical trial, and approximately 50% had participated in a trial. SCD individuals reported trusting their medical provider to provide them with information relevant to trials. Medical providers emphasized the individual, family, and health aspects of SCD that warrant additional investigation. Providers reported barriers encountered by patients as transportation, childcare support, infertility, and trust in the healthcare system. CONCLUSION: Lessons learned from the community have helped to influence recommendations for clinical trial design and key messaging development for the Cure Sickle Cell Initiative.

An assessment of the perceived benefits and challenges of participating in a practice-based research network.

BACKGROUND: A survey was conducted to describe the benefits of and challenges to practitioner participation in the Practitioners Engaged in Applied Research and Learning (PEARL) Network, a dental practice-based research network (PBRN). The results were compared with results from medical PBRNs across different tiers of participation (based on practitioner-investigators previous involvement with PEARL research protocols). METHODS: A 39-item web-based survey addressed the benefits of PBRN participation on three levels: individual/practitioner, practice (office), and community/professional. Participants were also asked to rate challenges to participation. RESULTS: A total of 153 of 216 PEARL practitioner-investigators participated, a response rate of 71%. The majority (70%) was male, with a median of 23 years in private practice. 'Means to stay informed of new developments in my profession' was considered a 'very important' benefit for nearly three-quarters of the sample (71%). 'Opportunity to improve clinical procedures' was considered as 'very important' by 73% of respondents. In terms of benefits related to the community and profession, 65% of respondents reported 'means to directly contribute to the evidence base of dental practice' as being 'very important'. 'Disruption in practice routine/clinical practice' was considered the most important challenge to participation. CONCLUSIONS: The benefits of and challenges to participation identified did not differ across tiers of participation and were similar to benefits identified by participants in medical PBRNs. The results of this study will help facilitate the design of future PBRN protocols to encourage greater participation by the profession. Results suggest that practitioners with similar interests could be recruited to collaborative studies between medicine and dentistry.

Cigarette smoking status and recurrent subjective health complaints among US school-aged adolescents.

BACKGROUND: Subjective health complaints are common among adolescents. There is evidence that girls are more likely to register complaints than boys. This study examines gender differences in the relationship between daily smoking and recurrent subjective health complaints in school-aged adolescents in the USA. METHODS: A cross-sectional design with a multistage probability sample was used to survey 13,339 middle and high school students (grades 6 through 10) with the US 2001-2002 Health Behavior in School-Aged Children Survey. RESULTS: Recurrent subjective health complaints were higher for adolescents who smoke daily and experiment with cigarettes than for those who never smoke. In logistic regression analyses, the odds of daily smoking increased twofold for both boys and girls who report recurrent irritability/bad temper. For girls, the odds of daily smoking were higher among those who reported recurrent headache, stomachache and backache compared with never smokers. For boys only recurrent backache and feeling dizzy were associated with increased odds of daily smoking. CONCLUSIONS: The relationship between recurrent subjective health complaints and daily smoking provides new insights into both conditions for school-aged adolescents. Findings from this study suggest different patterns of association between daily smoking and recurrent subjective health complaints occur for girls and boys. Further studies are needed to explore causes and treatment of daily smoking and recurrent health complaints among school-aged children.

Responsive parenting is associated with improved type 1 diabetes-related quality of life.

BACKGROUND: Improved quality of life is an important treatment goal for children and adolescents with type 1 diabetes. While previous research supports a relationship between family environment and quality of life, little research has addressed the relationship of parenting style constructs to quality of life in children with chronic disease. The present investigation assesses the relationship of parent responsiveness and demandingness with diabetes-related quality of life among children and adolescents with type 1 diabetes. METHODS: Baseline and 12-month follow-up self-report assessments were collected on a sample of 81 children with type 1 diabetes participating in an efficacy trial of a behavioural intervention to enhance adherence. The sample had a mean age of 13.3 years (SD=1.7) and duration of diabetes of 7.7 years (SD=3.7). Multiple regression analyses were conducted to determine the relationship of parent responsiveness and demandingness to diabetes-related quality of life at each time point. RESULTS: After adjusting for demographic and diabetes characteristics, as well as diabetes-specific parent-child behaviours, parent responsiveness was significantly associated with baseline diabetes-related quality of life (beta=0.23; P=0.04). This relationship was sustained at 12-month follow-up (beta=0.22; P=0.04) after adjusting for baseline quality of life and treatment group assignment, suggesting that parent responsiveness is associated with improved quality of life. CONCLUSIONS: Findings indicate the importance of a supportive and emotionally warm parenting style in promoting improved quality of life for children with type 1 diabetes. Appropriate parenting skills should be an element of diabetes family management health care.